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MDA Awards More than $7 Million in New Research Grants

New investment builds on recent breakthroughs with availability of three new neuromuscular disease drugs

CHICAGO, March 9, 2017 – The Muscular Dystrophy Association today announced the award of 29 new research and development grants totaling more than $7 million and targeted to impact the state of the science and accelerate the development of treatments for muscular dystrophy, ALS and related life-threatening diseases.

“MDA is excited to fund 29 new research projects at this time of unprecedented progress, with three drugs having been approved in the last six months for neuromuscular diseases — all developed with critical MDA support via grants like those we’re announcing today,” said MDA Executive Vice President and Chief Medical & Scientific Officer Valerie A. Cwik, M.D. “Each of the new projects under way now could lay the foundation for the next life-changing medical and scientific advances that will save and improve the lives of kids and adults fighting neuromuscular disease.”

For more than six decades, MDA has funded basic, translational and clinical research as it works to unlock the mechanisms of neuromuscular disease that will enable scientists and clinicians to develop therapies capable of changing lives. The power of MDA’s research program lies in its big-picture approach that leverages insights and information resulting from research in one disease area to inform and advance discoveries and breakthroughs in others. For the Winter 2017 round, MDA reviewed 187 grant applications and awarded grants to 29 leading scientists conducting innovative research targeted to make an impact across all the diseases in MDA’s program.

The latest round of research grants was approved by MDA’s Board of Directors following careful deliberations and analysis by MDA’s Research Advisory Committee, through which leading clinicians and scientists in volunteer roles oversee the peer-review process. This year, MDA is funding more than 150 different research projects around the world with a combined investment of nearly $50 million — dollars raised by MDA’s supporters.

Vital research and services dollars come from generous MDA partners and supporters who organize, and donate to, community fundraising programs such as Fill The Boot drives led by the International Association of Fire Fighters; retailers who do in-store promotions such as Lowe’s, CITGO Petroleum Corporation and Casey’s General Stores; and Harley-Davidson Motor Company enthusiasts who raise funds through dealer programs, rides and special events.

“MDA remains steadfast in our commitment to help make therapy options available to treat all the diseases under our umbrella as quickly as possible. Thanks to generous partners and donors who champion our work and our cause, we are moving more quickly than ever toward achieving that goal,” Cwik added. “We’re funding the most promising science in the neuromuscular disease space as we search for the solutions that will help MDA families live longer and grow stronger.”

Among the new MDA awards:

  • Mattia Quattrocelli, at the Center for Genetic Medicine, Northwestern University – Chicago, was awarded an MDA development grant totaling $180,000 over three years to study the effects of glucocorticoids on muscle repair and regeneration in Duchenne muscular dystrophy (DMD). The work, co-funded by the American Association of Neuromuscular & Electrodiagnostic Medicine Foundation for Research and Education (AANEM Foundation), is expected to help improve glucocorticoid-based treatment strategies in DMD.
  • James Novak, postdoctoral associate at Children’s Research Institute, Children’s National Health System in Washington, D.C., was awarded an MDA development grant totaling $180,000 over three years to examine why exon skipping drugs are most effective at getting to muscles that are actively undergoing repair. The work, co-funded by the Hearst Foundation, is expected to help optimize the effectiveness of exon skipping therapies in DMD.
  • Kleopas Kleopa, professor and senior consulting neurologist at the Cyprus Institute of Neurology and Genetics, Cyprus School of Molecular Medicine, in Nicosia, Cyprus, was awarded an MDA research grant totaling $119,999 over a period of two years to test whether gene therapy treatment after disease onset could lead to functional improvements in CMT1X, the second most common form of Charcot-Marie-Tooth disease (CMT). The grant is co-funded by the CMT Association.
  • Henry Kaminski, Meta A. Neumann Professor and Chair of the department of neurology at George Washington University in Washington, D.C., was awarded an MDA research grant totaling $367,187 over three years to test a therapeutic strategy in cell and rat models of myasthenia gravis (MG), with the intent to demonstrate the feasibility of the approach and then move to human clinical trials.
  • Thurman Wheeler, at Massachusetts General Hospital in Boston, was awarded an MDA research grant totaling $330,000 over three years to develop biomarkers in blood or urine that will reduce the need for muscle biopsies to measure disease progression and drug effectiveness in myotonic dystrophy (DM) and other neuromuscular disorders.

Winter 2017 Grants Materials

MDA has funded more than $1 billion in neuromuscular disease research since 1950, with an unprecedented three drugs approved in the last six months illustrating the vibrancy of its research program:

  • The U.S. Food and Drug Administration (FDA) in September 2016 granted accelerated approval to eteplirsen (brand name Exondys 51) to treat the most common childhood form of muscular dystrophy. Accelerated approval of the drug — the first disease-modifying drug for DMD — marked an important step forward in the development of therapies for neuromuscular diseases and marked an historic achievement for the entire DMD community. MDA has invested more than $210 million in DMD research, including support of the early development of Exondys 51.
  • In December, the FDA granted approval to nusinersen (brand name Spinraza), the first disease-modifying drug to treat the most common genetic cause of death in infants. Approval of the drug, which is used to treat kids and adults with all forms of spinal muscular atrophy (SMA) caused by a deficiency of SMN protein, represented a landmark achievement for the entire SMA community. MDA funded early-stage development of Spinraza and, since inception, has invested more than $45 million in SMA research.
  • The FDA in February approved deflazacort (brand name Emflaza) to treat DMD. Approval of the drug, which is labeled for use by people 5 years or older with DMD, regardless of genetic mutation, represents continued success for MDA and the entire DMD community. MDA has a long history of supporting research and clinical study into the effects of corticosteroids, including Emflaza, in DMD, and has invested more than $1.5 million in studies to determine drug effects, mechanism of action, side effects and best dosing regimen.

About the Muscular Dystrophy Association
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at mda.org.

Roxan Triolo Olivas
MDA Vice President Public Relations and Community Programs
(520) 529-5305